3. 脊髄性筋萎縮症 ［臨床試験数：75，薬物数：73（DrugBank：24），標的遺伝子数：49，標的パスウェイ数：75］
Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg Welander disease", "SMA IV"
The queries were searched in Public_title, Scientific_title, and Condition of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04089566 | February 26, 2020 | 4 November 2019 | Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Escalating Dose and Randomized, Controlled Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Drug: Nusinersen | Biogen | Not recruiting | N/A | N/A | All | 125 | Phase 2/Phase 3 | ||
| 2 | NCT04042025 | November 4, 2019 | 11 November 2019 | Long-term Follow-up Study of Patients Receiving Onasemnogene Abeparvovec-xioi | A Long-term Follow-up Study of Patients in the Clinical Trials for Spinal Muscular Atrophy Receiving AVXS-101 | Spinal Muscular Atrophy Type I;Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III | Biological: Onasemnogene Abeparvovec-xioi | AveXis, Inc. | Not recruiting | N/A | N/A | All | 308 | Phase 4 | United States;Australia;Belgium;Canada;France;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom | |
| 3 | NCT03779334 | August 8, 2019 | 4 November 2019 | A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Drug: Risdiplam | Hoffmann-La Roche | Recruiting | N/A | 6 Weeks | All | 25 | Phase 2 | United States;Australia;Belgium;Brazil;China;Italy;Poland;Russian Federation;Saudi Arabia | |
| 4 | NCT03988907 | June 18, 2019 | 4 November 2019 | A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants | A Phase I, 2-Part, Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics of Multiple Doses of Risdiplam and the Effect of Risdiplam on the Pharmacokinetics of Midazolam Following Oral Administration in Healthy Participants | Spinal Muscular Atrophy | Drug: Risdiplam;Drug: Midazolam | Hoffmann-La Roche | Not recruiting | 18 Years | 55 Years | All | 27 | Phase 1 | United States | |
| 5 | NCT03837184 | May 15, 2019 | 26 August 2019 | Single-Dose Gene Replacement Therapy Using for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies | Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion | Spinal Muscular Atrophy Type I | Biological: Onasemnogene Abeparvovec-xioi | AveXis, Inc. | PRA Health Sciences | Recruiting | N/A | 6 Months | All | 6 | Phase 3 | Japan;Korea, Republic of;Taiwan |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03921528 | April 22, 2019 | 2 September 2019 | An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy | Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients With Later-Onset Spinal Muscular Atrophy (TOPAZ) | Spinal Muscular Atrophy;Spinal Muscular Atrophy Type 3;Spinal Muscular Atrophy Type 2;SMA;Neuromuscular Diseases;Muscular Atrophy;Atrophy;Muscular Atrophy, Spinal;Neuromuscular Manifestations | Biological: SRK-015 | Scholar Rock, Inc. | Recruiting | 2 Years | 21 Years | All | 55 | Phase 2 | United States;Italy;Spain | |
| 7 | NCT03878030 | April 1, 2019 | 11 June 2019 | Effect of Nusinersen on Adults With Spinal Muscular Atrophy | Effect of Nusinersen on Motor Function in Adult Patients With Spinal Muscular Atrophy Types 2 and 3 | Adult Spinal Muscular Atrophy | Drug: nusinersen | Northwell Health | Recruiting | 18 Years | 60 Years | All | 40 | Phase 3 | United States | |
| 8 | NCT03819660 | March 7, 2019 | 8 April 2019 | Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 | Long Term Safety Study of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3 | Muscle Atrophy | Drug: Amifampridine Phosphate 10 MG Oral Tablet | Catalyst Pharmaceuticals, Inc. | Recruiting | 6 Years | 50 Years | All | 12 | Phase 2 | Italy | |
| 9 | NCT03648658 | February 18, 2019 | 11 March 2019 | Paracetamol Study in Patients With Low Muscle Mass | Pharmacokinetics and Safety of Treatment With Paracetamol in Children and Adults With Spinal Muscular Atrophy and Cerebral Palsy | SMA II;Cerebral Palsy | Drug: Paracetamol 120Mg/5mL Oral Suspension | Mette Cathrine Oerngreen | Elsass Foundation | Recruiting | 6 Years | 45 Years | All | 48 | Phase 4 | Denmark |
| 10 | NCT02876094 | January 29, 2019 | 26 August 2019 | Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy | A Pilot, Open-Label, Dose Response Study Investigating the Effect of Low-Dose Celecoxib on SMN2 in Patients With Spinal Muscular Atrophy (SMA) | Spinal Muscular Atrophy (SMA) | Drug: celecoxib | Hugh McMillan | Families of Spinal Muscular Atrophy;Gwendolyn Strong Foundation | Recruiting | 2 Years | 80 Years | All | 12 | Phase 2 | Canada |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT03781479 | January 21, 2019 | 4 February 2019 | Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients | A Randomized, Placebo-Controlled, Crossover Study to Evaluate the Safety and Efficacy of Amifampridine Phosphate in Ambulatory Patients With Spinal Muscular Atrophy (SMA) Type 3 | Muscular Atrophy, Spinal | Drug: Placebo Oral Tablet;Drug: Amifampridine Phosphate | Catalyst Pharmaceuticals, Inc. | Recruiting | 6 Years | 50 Years | All | 12 | Phase 2 | Italy | |
| 12 | EUCTR2018-002087-12-BE | 07/12/2018 | 30 April 2019 | A Study of Risdiplam in Infants with Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: LLT Classification code 10079419 Term: Spinal muscular atrophy pre-symptomatic System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F13 Pharmaceutical Form: Powder for oral solution INN or Proposed INN: Risdiplam CAS Number: 1825352-65-5 Current Sponsor code: RO7034067/F13 Other descriptive name: RO7034067 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 60- Pharmaceutical form of the placebo: Powder for oral solution Route of administration of the placebo: Oral use Product Name: RO7034067 Product Code: RO7034067/F12 Pharmaceutical Form: Powder for oral solution INN or Proposed INN: Risdiplam CAS Number: 1825352-65-5 Current Sponsor code: RO7034067/F12 Other descriptive name: RO7034067 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- | F. Hoffmann-La Roche Ltd | Authorised | Female: yes Male: yes | 25 | Phase 2 | United States;Saudi Arabia;Poland;Brazil;Belgium;Australia;Russian Federation;Italy;China | |||
| 13 | EUCTR2018-002295-40-DK | 11/09/2018 | 24 September 2018 | Is treatment with the painkiller paracetamol safe in patients with spinal muscular atrophy and cerebral palsy? | Pharmacokinetics and safety of treatment with paracetamol in children and adults with spinal muscular atrophy and cerebral palsy - Paracetamol study in patients with low muscle mass | Spinal muscular atrophy type II (SMA II) Cerebral palsy (CP) MedDRA version: 20.1 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.1 Level: PT Classification code 10008129 Term: Cerebral palsy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Pharmaceutical Form: Oral suspension INN or Proposed INN: Paracetamol Other descriptive name: PARACETAMOL Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 24- | Copenhagen Neuromuscular Center | Authorised | Female: yes Male: yes | 24 | Phase 1;Phase 4 | Denmark | |||
| 14 | NCT03461289 | August 29, 2018 | 7 October 2019 | Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 | European, Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion | SMA | Biological: Onasemnogene Abeparvovec-xioi | AveXis, Inc. | Not recruiting | N/A | 6 Months | All | 33 | Phase 3 | Belgium;Italy;United Kingdom;France;Germany;Netherlands;Spain;Sweden | |
| 15 | JPRN-JapicCTI-183891 | 01/6/2018 | 16 July 2019 | Investigate Safety, Tolerability, PK, PD and Efficacy of RO7034067 in Infants With Type1 Spinal Muscular Atrophy (FIREFISH) | A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Spinal Muscular Atrophy | Spinal Muscular Atrophy | Intervention name : RO7034067 INN of the intervention : risdiplam Dosage And administration of the intervention : po | CHUGAI PHARMACEUTICAL CO., LTD. | Not Recruiting | BOTH | 40 | Phase 2 | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT03505099 | April 10, 2018 | 26 August 2019 | Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants With Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy With Multiple Copies of SMN2 | Spinal Muscular Atrophy | Biological: onasemnogene abeparvovec-xioi | AveXis, Inc. | PRA Health Sciences | Recruiting | N/A | 42 Days | All | 27 | Phase 3 | United States;Australia;Belgium;Canada;Germany;Israel;Italy;Japan;Korea, Republic of;Spain;Taiwan;United Kingdom |
| 17 | EUCTR2018-004383-65-NL | 30 April 2019 | A clinical study of a new possible treatment in patients with type 2 or 3 Spinal Muscular Atrophy | Phase 2 Active Treatment Study to Evaluate the Efficacy and Safety of SRK-015 in Patients with Later-Onset Spinal Muscular Atrophy - The TOPAZ study | Later Onset Spinal Muscular Atrophy (SMA) MedDRA version: 20.0 Level: LLT Classification code 10079415 Term: Spinal muscular atrophy type III System Organ Class: 100000004850 MedDRA version: 20.0 Level: LLT Classification code 10079416 Term: Spinal muscular atrophy type II System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: SRK-015 Pharmaceutical Form: Solution for injection/infusion INN or Proposed INN: SRK-015 Current Sponsor code: SRK-015 Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- Product Name: SRK-015 Pharmaceutical Form: Solution for injection/infusion INN or Proposed INN: SRK-015 Current Sponsor code: SRK-015 Other descriptive name: HUMAN ANTI-PROMYOSTATIN MONOCLONAL ANTIBODY Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 50- | Scholar Rock, Inc. | Not Available | Female: yes Male: yes | 55 | Phase 2 | United States;Canada;Spain;Germany;Netherlands;Italy | ||||
| 18 | NCT03381729 | December 14, 2017 | 26 August 2019 | Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy | Phase I, Open-Label, Dose Comparison Study of AVXS-101 for Sitting But Non-ambulatory Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Biological: Onasemnogene Abeparvovec-xioi | AveXis, Inc. | Recruiting | 6 Months | 60 Months | All | 51 | Phase 1 | United States | |
| 19 | JPRN-JapicCTI-173722 | 01/12/2017 | 16 July 2019 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Participants (Sunfish) | A Two-Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients | Spinal Muscular Atrophy | Intervention name : RO7034067 INN of the intervention : risdiplam Dosage And administration of the intervention : po Control intervention name : Placebo Dosage And administration of the control intervention : po | CHUGAI PHARMACEUTICAL CO., LTD. | Not Recruiting | 2 | 25 | BOTH | 168 | Phase 2 | ||
| 20 | NCT03306277 | October 24, 2017 | 26 August 2019 | Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 | Phase 3, Open-Label, Single-Arm, Single-Dose Gene Replacement Therapy Clinical Trial for Patients With Spinal Muscular Atrophy Type 1 With One or Two SMN2 Copies Delivering AVXS-101 by Intravenous Infusion | SMA - Spinal Muscular Atrophy;Gene Therapy | Biological: Onasemnogene Abeparvovec-xioi | AveXis, Inc. | Not recruiting | N/A | 180 Days | All | 20 | Phase 3 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT03421977 | August 15, 2017 | 26 August 2019 | Long-Term Follow-up Study for Patients From AVXS-101-CL-101 | A Long Term Follow up Safety Study of Patients in the AVXS-101-CL-101 Gene Replacement Therapy Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS 101 | Spinal Muscular Atrophy 1 | Biological: Onasemnogene Abeparvovec-xioi | AveXis, Inc. | Recruiting | N/A | N/A | All | 15 | N/A | United States | |
| 22 | NCT03032172 | March 3, 2017 | 4 November 2019 | A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy | An Open-Label Study to Investigate the Safety, Tolerability, and Pharmacokinetics/Pharmacodynamics of RO7034067 in Adult and Pediatric Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Risdiplam | Hoffmann-La Roche | Recruiting | 6 Months | 60 Years | All | 180 | Phase 2 | United States;Belgium;France;Germany;Italy;Netherlands;Poland;Switzerland;United Kingdom | |
| 23 | EUCTR2016-000778-40-DE | 05/01/2017 | 5 June 2018 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants with Type1 Spinal Muscular Atrophy | A TWO PART SEAMLESS, OPEN-LABEL, MULTICENTER STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN INFANTS WITH TYPE1 SPINAL MUSCULAR ATROPHY | Type 1 Spinal Muscular Atrophy (SMA) MedDRA version: 20.1 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F06 with solvent (RO7034067/F08) Pharmaceutical Form: Powder and solvent for oral solution INN or Proposed INN: n/a Current Sponsor code: RO7034067/F06 Other descriptive name: RO7034067 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- Product Name: RO7034067 Product Code: RO7034067/F07 with solvent (RO7034067/F09) Pharmaceutical Form: Powder and solvent for oral solution INN or Proposed INN: n/a Current Sponsor code: RO7034067/F07 Other descriptive name: RO7034067 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 120- Product Name: RO7034067 Product Code: RO7034067 / F12 Pharmaceutical Form: Powder for oral solution INN or Proposed INN: n/a Current Sponsor code: RO7034067/F12 Other descriptive name: RO7034067 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- Product Name: RO7034067 Product Code: RO7034067 / F13 Pharmaceutical Form: Powder for oral solution INN or Proposed INN: n/a Current Sponsor code: RO7034067 / F13 Other descriptive name: RO7034067 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 60- | F. Hoffmann-La Roche Ltd | Authorised | Female: yes Male: yes | 64 | Phase 2 | Serbia;United States;Saudi Arabia;Spain;Ukraine;Lebanon;Turkey;Russian Federation;Italy;Switzerland;United Kingdom;France;Canada;Poland;Belgium;Brazil;Croatia;Germany;Japan;China | |||
| 24 | EUCTR2017-004087-35-DE | 19 November 2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 Pharmaceutical Form: Infusion INN or Proposed INN: onasemnogene abeparvovec CAS Number: 1922968-73-7 Current Sponsor code: AVXS-101 Concentration unit: vector genomes (vg)/mL Concentration type: range Concentration number: 20000000000000-60000000000000 | AveXis, Inc. | Not Available | Female: yes Male: yes | 27 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Germany;Netherlands;Japan | ||||
| 25 | EUCTR2017-004087-35-NL | 23 July 2018 | Single dose gene replacement therapy clinical trial for infants with genetically diagnosed and pre-symptomatic Spinal Muscular Atrophy | A Global Study of a Single, One-Time Dose of AVXS-101 Delivered to Infants with Genetically Diagnosed and Pre-symptomatic Spinal Muscular Atrophy with Multiple Copies of SMN2 - SPR1NT | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: AVXS-101 (previously known as scAAV9.CB.SMN) Product Code: AVXS-101 Pharmaceutical Form: Infusion INN or Proposed INN: onasemnogene abeparvovec CAS Number: 1922968-73-7 Current Sponsor code: AVXS-101 Concentration unit: vector genomes (vg)/mL Concentration type: range Concentration number: 20000000000000-60000000000000 | AveXis, Inc. | Not Available | Female: yes Male: yes | 44 | Phase 3 | United States;Taiwan;Spain;Korea, Democratic People's Republic of;Israel;United Kingdom;Italy;Canada;Belgium;Australia;Netherlands;Germany;Japan | ||||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 26 | NCT02913482 | December 24, 2016 | 16 September 2019 | Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy | A Two Part Seamless, Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Infants With Type 1 Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Drug: Risdiplam | Hoffmann-La Roche | Not recruiting | 1 Month | 7 Months | All | 48 | Phase 2/Phase 3 | United States;Belgium;Brazil;China;Croatia;France;Italy;Japan;Poland;Russian Federation;Saudi Arabia;Serbia;Spain;Switzerland;Turkey;Australia;Germany;Taiwan | |
| 27 | NCT02908685 | October 20, 2016 | 16 September 2019 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants | A Two Part Seamless, Multi-Center Randomized, Placebo-Controlled, Double-Blind Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients | Muscular Atrophy, Spinal | Drug: Placebo;Drug: Risdiplam | Hoffmann-La Roche | Not recruiting | 2 Years | 25 Years | All | 231 | Phase 2/Phase 3 | United States;Belgium;Brazil;Canada;China;Croatia;France;Germany;Italy;Japan;Poland;Russian Federation;Serbia;Spain;Turkey;Argentina;Australia;Sweden;Switzerland;Taiwan;United Kingdom | |
| 28 | EUCTR2016-000750-35-IT | 29/08/2016 | 20 February 2017 | A Study to investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics And Efficacy of RO7034067 in Type 2 and 3 Spinal Muscular Atrophy Patients | A TWO-PART SEAMLESS, MULTI-CENTER RANDOMIZED, PLACEBO-CONTROLLED, DOUBLE BLIND STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS AND EFFICACY OF RO7034067 IN TYPE 2 AND 3 SPINAL MUSCULAR ATROPHY PATIENTS. | Spinal Muscular Atrophy (SMA) MedDRA version: 19.0 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: RO7034067 Product Code: RO7034067/F06 with solvent (RO7034067/F08) Pharmaceutical Form: Powder and solvent for oral solution INN or Proposed INN: n.a Current Sponsor code: RO7034067/F06 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 20- Pharmaceutical form of the placebo: Powder and solvent for oral solution Route of administration of the placebo: Oral use Product Name: RO7034067 Product Code: RO7034067/F07 with solvent (RO7034067/F09) Pharmaceutical Form: Powder and solvent for oral solution INN or Proposed INN: n.a Current Sponsor code: RO7034067/F07 Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 120- Pharmaceutical form of the placebo: Powder and solvent for oral solution Route of administration of the placebo: Oral use | F. Hoffmann-La Roche Ltd | Authorised | Female: yes Male: yes | 186 | Phase 2 | United States;Spain;Turkey;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Australia;Germany;Netherlands;Sweden | |||
| 29 | JPRN-JMA-IIA00259 | 16/08/2016 | 2 April 2019 | Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Continuous administration trial | Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Continuous administration trial | spinal muscular atrophy | Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA 12.5mg/kg or 25mg/kg is to be taken once a day after supper.. | Institute of Medical Genetics, Tokyo Women's Medical University | Recruiting | >=1 YEARS | <8 YEARS | BOTH | 28 | Phase 2B | Japan | |
| 30 | EUCTR2015-001870-16-DE | 08/03/2016 | 30 April 2019 | An extension study for patients with Spinal Muscular Atrophy who participated to the previous ISIS 396443 studies | An Open-label Extension Study for Patients with Spinal Muscular Atrophy who Previously Participated in Investigational Studies of ISIS 396443 | Spinal Muscular Atrophy (SMA) MedDRA version: 20.1 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 Pharmaceutical Form: Solution for injection INN or Proposed INN: Nusinersen CAS Number: 1258984-36-9 Current Sponsor code: ISIS 396443 (BIIB058) Other descriptive name: ISIS 396443 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 2.4- | Biogen Idec Research Limited | Authorised | Female: yes Male: yes | 292 | Phase 3 | United States;Hong Kong;Spain;Turkey;United Kingdom;Italy;France;Canada;Belgium;Australia;Germany;Japan;Korea, Republic of;Sweden | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 31 | JPRN-JMA-IIA00231 | 29/01/2016 | 2 April 2019 | Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Confirmatory Trial | Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy : Confirmatory Trial | spinal muscular atrophy | Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA 12.5mg/kg or 25mg/kg is to be taken once a day after supper.. Control intervention1:Placebo, Dose form:GRANULES, Route of administration:ORAL, Intended dose regimen:Placebo is to be taken once a day after supper.. | Institute of Medical Genetics, Tokyo Women's Medical University | Not Recruiting | >=1 YEARS | <7 YEARS | BOTH | 28 | Phase 2B | Japan | |
| 32 | NCT02628743 | January 20, 2016 | 18 March 2019 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA) | Multicenter, Open-Label, Single-Arm Study to Evaluate Long-Term Safety, Tolerability, and Effectiveness of 10 mg/kg BID Olesoxime in Patients With Spinal Muscular Atrophy | Muscular Atrophy, Spinal | Drug: Olesoxime | Hoffmann-La Roche | Not recruiting | N/A | N/A | All | 131 | Phase 2 | Belgium;France;Germany;Italy;Netherlands;Poland;United Kingdom | |
| 33 | NCT02633709 | January 7, 2016 | 9 October 2018 | A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers | A Single-Center, Randomized, Investigator/Subject-Blind, Adaptive Single-Ascending-Dose(SAD), Placebo-Controlled, Parallel Study to Investigate the Safety, Tolerability, Pharmacokinetics (Including the Effect of Food and the Effect of Itraconazole on the Pharmacokinetics of a Single Oral Dose of RO7034067), and Pharmacodynamics of RO7034067 Following Oral Administration in Healthy Subjects | Spinal Muscular Atrophy | Drug: Itraconazole;Other: Placebo;Drug: Risdiplam | Hoffmann-La Roche | Not recruiting | 18 Years | 45 Years | Male | 33 | Phase 1 | Netherlands | |
| 34 | NCT02644668 | December 2015 | 18 March 2019 | A Study of CK-2127107 in Patients With Spinal Muscular Atrophy | A Phase 2, Double-Blind, Randomized, Placebo-Controlled, Multiple Dose Study of CK-2127107 in Two Ascending Dose Cohorts of Patients With Spinal Muscular Atrophy (SMA) | Spinal Muscular Atrophy | Drug: CK-2127107 150 mg;Drug: Placebo;Drug: CK-2127107 450 mg | Cytokinetics | Astellas Pharma Global Development, Inc. | Not recruiting | 12 Years | N/A | All | 70 | Phase 2 | United States;Canada |
| 35 | NCT02941328 | December 2015 | 12 February 2018 | SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4 | A Phase II, Mono-center, Placebo-controlled, Double-blind, Crossover Trial to Investigate Effect and Efficacy of Pyridostigmine in Dutch Patients With Spinal Muscular Atrophy Types 2, 3 and 4 | Spinal Muscular Atrophy;SMA;Kugelberg-Welander Disease | Drug: Pyridostigmine;Drug: Placebo | UMC Utrecht | Not recruiting | 12 Years | N/A | All | 39 | Phase 2 | Netherlands | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 36 | EUCTR2015-001589-25-GB | 19/11/2015 | 11 December 2017 | A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients with Spinal Muscular Atrophy | Multicenter, open-label, single-arm study to evaluate long term safety, tolerability, and effectiveness of 10mg/kg BID olesoxime in patients with Spinal Muscular Atrophy - OLEOS | Spinal Muscular Atrophy MedDRA version: 20.1 Level: LLT Classification code 10051203 Term: Spinal muscular atrophy congenital System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: Olesoxime Product Code: RO7090919 Pharmaceutical Form: Powder and solvent for oral suspension INN or Proposed INN: OLESOXIME CAS Number: 22033-87-0 Current Sponsor code: RO7090919/F01 Concentration unit: g gram(s) Concentration type: equal Concentration number: 7.5- INN or Proposed INN: SESAME OIL, REFINED Current Sponsor code: RO7090919/F02 Other descriptive name: SESAME OIL, REFINED Concentration unit: ml millilitre(s) Concentration type: equal Concentration number: 67.1- | F. Hoffmann-La Roche Ltd | Authorised | Female: yes Male: yes | 173 | Phase 2;Phase 3 | Belgium;Netherlands;Germany;Italy;United Kingdom | |||
| 37 | NCT02594124 | November 4, 2015 | 4 November 2019 | A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies. | An Open-Label Extension Study for Patients With Spinal Muscular Atrophy Who Previously Participated in Investigational Studies of ISIS 396443 | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | Not recruiting | N/A | N/A | All | 292 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Taiwan | |
| 38 | EUCTR2014-003657-33-DE | 21/09/2015 | 14 January 2019 | A Study to assess the safety and tolerability of ISIS 396443 in participants with spinal muscular atrophy (SMA). | A phase 2, randomized, double-blind, sham-procedure controlled study to assess the safety and tolerability and explore the efficacy of ISIS 396443 (BIIB058) administered intrathecally in subjects with spinal muscular atrophy who are not eligible to participate in the clinical studies ISIS 396443-CS3B or ISIS 396443-CS4 - EMBRACE | Spinal Muscular Atrophy MedDRA version: 20.1 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05] | Trade Name: Spinraza Product Name: Survival of Motor Neuron 2 (SMN2) Splicing Modulator Antisense Oligonucleotide Product Code: ISIS 396443 (BIIB058) Pharmaceutical Form: Solution for injection INN or Proposed INN: Nusinersen CAS Number: 1258984-36-9 Current Sponsor code: ISIS 396443 (BIIB058) Other descriptive name: ISIS 396443 (BIIB058) Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 2.4- | Biogen Idec Research Limited | Not Recruiting | Female: yes Male: yes | 16 | Phase 2 | United States;Germany;United Kingdom | |||
| 39 | NCT02855112 | June 2015 | 15 August 2016 | Allogeneic Adipose Derived Stem Cells for Werdnig Hoffman Patients | The Effectiveness of Allogeneic Adipose Derived Mesenchymal Stem Cells (ADMSCs) in the Phenotypic Changes of Werdnig Hoffman Patients | Infantile Spinal Muscular Atrophy, Type I [Werdnig- Hoffman] | Biological: Adipose derived mesenchymal stem cell | Tehran University of Medical Sciences | Recruiting | 5 Months | 12 Months | Both | 10 | Phase 1/Phase 2 | Iran, Islamic Republic of | |
| 40 | NCT02386553 | May 20, 2015 | 4 November 2019 | A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy | An Open-Label Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Subjects With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy. | Spinal Muscular Atrophy | Drug: Nusinersen | Biogen | Not recruiting | N/A | 6 Weeks | All | 25 | Phase 2 | United States;Australia;Germany;Italy;Qatar;Taiwan;Turkey;Argentina;Israel;United Kingdom | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 41 | EUCTR2011-004369-34-NL | 22/04/2015 | 30 April 2019 | SPACE trial SMA and Pyridostigmine in Adults and Children; Experimental trial to assess effect of pyridostigmine compared to placebo in patients with spinal muscular atrophy types 2, 3 and 4 | SPACE trial SMA and Pyridostigmine in Adults and Children; Efficacy trial Phase II, mono-center, doubleblind, placebo-controlled, crossover trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2, 3 and 4 - SPACE trial | Proximal spinal muscular atrophy (SMA) is characterized by weakness of predominantly axial and proximal muscle groups and is caused by homozygous deletion of the survival motor neuron 1 (SMN1)-gene. There are 4 SMA types (type 1-4), with a descending order of severity. Age at onset and achieved motor milestones are the characteristics to define severity. Treatment of SMA is exclusively supportive. MedDRA version: 17.1 Level: LLT Classification code 10068209 Term: Spinal muscular atrophy adult onset System Organ Class: 100000004850 MedDRA version: 17.1 Level: LLT Classification code 10051203 Term: Spinal muscular atrophy congenital System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Trade Name: pyridostigmine bromide Product Name: Mestinon Product Code: RVG 03820 Pharmaceutical Form: Tablet Pharmaceutical form of the placebo: Tablet Route of administration of the placebo: Oral use | Universtiy Medical Center Utrecht | Not Recruiting | Female: yes Male: yes | 45 | Phase 2 | Netherlands | |||
| 42 | NCT02268552 | April 2, 2015 | 4 November 2019 | An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) | An Open Label Multi-part First-in-human Study of Oral LMI070 in Infants With Type 1 Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: branaplam | Novartis Pharmaceuticals | Not recruiting | N/A | 182 Days | All | 39 | Phase 1/Phase 2 | Belgium;Bulgaria;Denmark;Germany;Italy;Poland;Russian Federation;Czechia;Hungary;Netherlands | |
| 43 | EUCTR2014-002053-19-IT | 01/04/2015 | 28 February 2019 | Clinical trial of LMI070 given my mouth to Type I SMA infant patients. | An open-label multi-part first-in-human study of oral LMI070 in infants with Type 1 spinal muscular atrophy - CLMI070X2201 | Spinal Muscular Atrophy;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Name: NA Product Code: LMI070 Pharmaceutical Form: Oral solution INN or Proposed INN: NA CAS Number: NA Current Sponsor code: LMI070 Other descriptive name: NA Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 3.5- | NOVARTIS FARMA S.p.A. | Authorised | Female: yes Male: yes | 22 | Phase 1;Phase 2 | United States;Hungary;Czech Republic;European Union;Poland;Belgium;Denmark;Bulgaria;Netherlands;Germany;Italy | |||
| 44 | NCT02292537 | November 2014 | 16 December 2017 | A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) | A Phase 3, Randomized, Double-blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Later-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: nusinersen;Procedure: Sham procedure | Biogen | Not recruiting | 2 Years | 12 Years | All | 126 | Phase 3 | United States;Canada;France;Germany;Hong Kong;Italy;Japan;Korea, Republic of;Spain;Sweden;United Kingdom | |
| 45 | EUCTR2014-002246-41-IT | 23/10/2014 | 31 January 2017 | A Study of RO6885247 in Adult and Pediatric Patients with Spinal Muscular Atrophy (MOONFISH) | A MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE-DOSE STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF RO6885247 FOLLOWING 12 WEEKS OF TREATMENT IN ADULT AND PEDIATRIC PATIENTS WITH SPINAL MUSCULAR ATROPHY (MOONFISH) - MOONFISH | Spinal Muscular Atrophy MedDRA version: 17.0 Level: PT Classification code 10041582 Term: Spinal muscular atrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Nervous System Diseases [C10] | Product Code: RO6885247/F03 Pharmaceutical Form: Powder for oral solution INN or Proposed INN: n.a. CAS Number: 1449598-06-4 Current Sponsor code: RO6885247 Other descriptive name: n.a. Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 80- Pharmaceutical form of the placebo: Oral solution Route of administration of the placebo: Oral use | F. Hoffmann-La Roche Ltd. | Not Recruiting | Female: yes Male: yes | 48 | Phase 1 | United States;Spain;Netherlands;United Kingdom;Switzerland;Italy | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 46 | NCT02193074 | August 19, 2014 | 16 December 2017 | A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy | A Phase 3, Randomized, Double-Blind, Sham-Procedure Controlled Study to Assess the Clinical Efficacy and Safety of ISIS 396443 Administered Intrathecally in Patients With Infantile-onset Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: nusinersen;Procedure: Sham procedure | Biogen | Not recruiting | N/A | 210 Days | All | 122 | Phase 3 | United States;Australia;Belgium;Canada;France;Germany;Italy;Japan;Korea, Republic of;Spain;Sweden;Turkey;United Kingdom;Hong Kong;Taiwan | |
| 47 | JPRN-JMA-IIA00190 | 22/07/2014 | 2 April 2019 | Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy | Multicenter cooperative and investigator initiated clinical trial using valproic acid in childhood onset spinal muscular atrophy | spinal muscular atrophy | Intervention type:DRUG. Intervention1:SMART, Dose form:GRANULES, Route of administration:ORAL, intended dose regimen:VPA is to be taken once a day after supper. Administration of VPA starts with an initial dosage (standard dose: 12.5 mg / kg) for 4 weeks. The dosage then increases to a maintenance dosage (standard dose: 25 mg/kg) from the 5th week to the 12th week. Administration, however, could be increased to an additional 50 mg/day in case of a blood concentration of VPA below 50 mcg/mL From the 13th week, administered doses are to be decreased (standard dose: 12.5 mg/kg) and continued to the 14th week. . Control intervention1:No. | Institute of Medical Genetics, Tokyo Women's Medical University | Not Recruiting | No Limit | <8 YEARS | BOTH | 13 | Phase 2A | Japan | |
| 48 | NCT02227823 | July 2014 | 19 February 2015 | Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3 | Safety and Efficacy Study of Anti-cholinesterase Therapy on the Motor Functions in Patients With Spinal Muscular Atrophy Type 3. | Spinal Muscular Atrophy Type 3 | Drug: Pyridostigmine Bromide | Centre Hospitalier Régional de la Citadelle | Recruiting | 6 Years | N/A | Both | 20 | Phase 2 | Belgium | |
| 49 | NCT02122952 | May 5, 2014 | 12 March 2018 | Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 | Phase I Gene Transfer Clinical Trial for Spinal Muscular Atrophy Type 1 Delivering AVXS-101 | Spinal Muscular Atrophy 1 | Biological: AVXS-101 | AveXis, Inc. | Not recruiting | N/A | 6 Months | All | 15 | Phase 1 | United States | |
| 50 | NCT02052791 | January 2014 | 16 December 2017 | An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246) | An Open-label Study to Assess the Safety and Tolerability of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in 396443-CS2 or 396443-CS10 | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | Not recruiting | N/A | N/A | All | 47 | Phase 1 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 51 | NCT01671384 | August 2013 | 18 January 2016 | Valproate and Levocarnitine in Children With Spinal Muscular Atrophy | Randomized Placebo Controlled Trial of Valproate and Levocarnitine in Children With Spinal Muscular Atrophy Aged 2-15 Years | Spinal Muscular Atrophy | Drug: Valproate, Levocarnitine;Drug: Placebo | All India Institute of Medical Sciences, New Delhi | Recruiting | 2 Years | 15 Years | Both | 60 | Phase 3 | India | |
| 52 | NCT01839656 | May 31, 2013 | 11 June 2018 | A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) | A Study to Assess the Efficacy, Safety, Tolerability, and Pharmacokinetics of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Infantile-Onset Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | Not recruiting | N/A | 210 Days | All | 20 | Phase 2 | United States;Canada | |
| 53 | NCT01780246 | January 2013 | 16 December 2017 | An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701) | An Open-label Study to Assess the Safety and Tolerability of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | Not recruiting | 2 Years | 15 Years | All | 18 | Phase 1 | United States | |
| 54 | NCT01703988 | October 2012 | 16 December 2017 | An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy | An Open-Label, Dose Escalation Study to Assess the Safety, Tolerability and Dose-Range Finding of Multiple Doses of ISIS 396443 Delivered Intrathecally to Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Nusinersen | Biogen | Not recruiting | 2 Years | 15 Years | All | 34 | Phase 1/Phase 2 | United States | |
| 55 | NCT01645787 | June 2012 | 30 May 2016 | Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients | Columbia SMA Project: 4-AP as a Potential SMA Therapeutic Agent and Biological Mechanisms of Action | Spinal Muscular Atrophy | Drug: 4-aminopyridine;Drug: Placebo | Columbia University | Not recruiting | 18 Years | 50 Years | Both | 11 | Phase 2/Phase 3 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 56 | NCT01494701 | November 30, 2011 | 16 December 2017 | An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) | An Open-label, Escalating Dose Study to Assess the Safety, Tolerability and Dose-range Finding of a Single Intrathecal Dose of ISIS 396443 in Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: nusinersen | Biogen | Not recruiting | 2 Years | 14 Years | All | 28 | Phase 1 | United States | |
| 57 | NCT01422200 | August 2011 | 19 February 2015 | Flu Vaccine Study in Neuromuscular Patients 2011 | Comparison of the Immunogenicity of Intramuscular Versus Subcutaneous Administration of Trivalent Inactivated Influenza Vaccine in Individuals With Neuromuscular Diseases | Duchenne Muscular Dystrophy;Spinal Muscular Atrophy;Congenital Muscular Dystrophy | Biological: 2011-2012 seasonal flu vaccine | Children's Hospital Medical Center, Cincinnati | Recruiting | 3 Years | 35 Years | Both | 30 | N/A | United States | |
| 58 | ChiCTR-TRC-10001093 | 2010-10-01 | 18 April 2017 | Rat nerve growth factor injection in the treatment of children with spinal muscular atrophy: a randomized controlled trial | Rat nerve growth factor injection in the treatment of children with spinal muscular atrophy: a randomized controlled trial | children with spinal muscular atrophy | A:Rat nerve growth factor injection for six monthes,;B:Rat nerve growth factor injection for six monthes,; | PLA General Hospital | Not Recruiting | 3 | 8 | Both | A:20;B:20; | Post-market | China | |
| 59 | EUCTR2010-020386-24-FR | 2 October 2017 | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Phase II, multicenter, randomized, adaptive, double-blind, placebo controlled study to assess safety and efficacy of olesoxime (TRO19622) in 3-25 year old Spinal Muscular Atrophy (SMA) patients. | Spinal Muscular Atrophy (type 2 or type 3 non ambulant patients aged 3-25 years) MedDRA version: 12.1 Level: LLT Classification code 10041582 Term: Spinal muscular atrophy | Product Name: OLESOXIME Product Code: TRO19622 Pharmaceutical Form: Oral suspension INN or Proposed INN: olesoxime CAS Number: 2203-87-0 Current Sponsor code: TRO19622 Other descriptive name: 4-cholesten-3-one, oxime Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 100- Pharmaceutical form of the placebo: Oral suspension Route of administration of the placebo: Oral use | TROPHOS SA | Not Available | Female: yes Male: yes | Phase 2 | France;Belgium;Germany;Netherlands;United Kingdom;Italy | |||||
| 60 | EUCTR2008-003915-11-DE | 24/09/2009 | 17 December 2012 | Phase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I) - CARNI-VAL Type I | Phase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I) - CARNI-VAL Type I | Spinal Muscular Atrophy Type I in infants MedDRA version: 9.1 Level: LLT Classification code 10051203 Term: Spinal muscular atrophy congenital | Trade Name: Orfiril Saft Pharmaceutical Form: Oral solution INN or Proposed INN: VALPROATE SODIUM CAS Number: 1069665 Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 60- Trade Name: Biocarn Pharmaceutical Form: Oral solution INN or Proposed INN: LEVOCARNITINE CAS Number: 541151 Concentration unit: g/ml gram(s)/millilitre Concentration type: equal Concentration number: 0.303030- | University of Utah | Not Recruiting | Female: yes Male: yes | 36 | Phase 1/2 | Germany | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 61 | EUCTR2007-001088-32-IT | 29/07/2008 | 19 March 2012 | A PHASE II RANDOMIZED, DOUBLE-BLIND STUDY VS. PLACEBO FOR THE EVALUATION OF EFFICACY AND TOLERABILITY OF SALBUTAMOL ADMINISTERED BY ORAL ROUTE IN PATIENTS AFFECTED BY SPINAL MUSCULAR ATHROPHY. - ND | A PHASE II RANDOMIZED, DOUBLE-BLIND STUDY VS. PLACEBO FOR THE EVALUATION OF EFFICACY AND TOLERABILITY OF SALBUTAMOL ADMINISTERED BY ORAL ROUTE IN PATIENTS AFFECTED BY SPINAL MUSCULAR ATHROPHY. - ND | Patients affected by SMA MedDRA version: 9.1 Level: LLT Classification code 10041582 Term: Spinal muscular atrophy | Trade Name: VOLMAX*30CPR 4MG R.P. Pharmaceutical Form: Modified-release tablet INN or Proposed INN: Salbutamol Concentration unit: mg milligram(s) Concentration type: equal Concentration number: 4- Pharmaceutical form of the placebo: Modified-release tablet Route of administration of the placebo: Oral use | ISTITUTO NEUROLOGICO CARLO BESTA | Not Recruiting | Female: yes Male: yes | Phase 2 | Italy | ||||
| 62 | NCT00661453 | April 2008 | 19 October 2017 | CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I | Phase I/II Trial of Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy Type I (CARNI-VAL Type I) | Spinal Muscular Atrophy Type I | Drug: Valproic Acid and Levocarnitine | University of Utah | Families of Spinal Muscular Atrophy;Leadiant Biosciences, Inc. | Not recruiting | N/A | 12 Months | All | 40 | Phase 1/Phase 2 | United States;Canada;Germany |
| 63 | NCT00439218 | January 2008 | 19 October 2017 | Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I | Phase I/IIa Clinical Trial of Sodium Phenylbutyrate in Pediatric Subjects With Type I Spinal Muscular Atrophy | Spinal Muscular Atrophy Type I | Drug: sodium phenylbutyrate | Westat | National Institute of Neurological Disorders and Stroke (NINDS) | Not recruiting | 2 Months | 48 Months | All | 5 | Phase 1/Phase 2 | United States |
| 64 | NCT00439569 | January 2008 | 19 October 2017 | Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III | Phase I/IIa Clinical Trial of Sodium Phenylbutyrate in Pediatric Subjects With Type II/III Spinal Muscular Atrophy | Spinal Muscular Atrophy Type II;Spinal Muscular Atrophy Type III | Drug: sodium phenylbutyrate | Westat | National Institute of Neurological Disorders and Stroke (NINDS) | Not recruiting | 2 Years | 11 Years | All | 9 | Phase 1/Phase 2 | United States |
| 65 | NCT00481013 | July 2007 | 9 January 2017 | Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy | Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study | Spinal Muscular Atrophy | Drug: Valproic Acid (VPA);Drug: Placebo | University of Utah | Families of Spinal Muscular Atrophy;Abbott | Not recruiting | 18 Years | 60 Years | Both | 33 | Phase 2 | United States |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 66 | NCT00528268 | July 2007 | 19 October 2017 | Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy | Prospective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Sodium phenylbutyrate (NaPB) | University of Utah | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | Not recruiting | N/A | 6 Months | All | 14 | Phase 1/Phase 2 | United States |
| 67 | NCT00485511 | June 2007 | 19 February 2015 | A Trial of Hydroxyurea in Spinal Muscular Atrophy | A Randomized, Double-Blind, Placebo-Controlled Trial of Hydroxyurea in Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Hydroxyurea | Kaohsiung Medical University Chung-Ho Memorial Hospital | Not recruiting | 4 Years | N/A | Both | Phase 2/Phase 3 | Taiwan | ||
| 68 | EUCTR2006-006845-14-FR | 20/03/2007 | 19 March 2012 | Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients. | Open-label Phase 1b, Dose-ranged, Single and Multiple Dose Study to assess Safety and Pharmacokinetics of TRO19622 in 6-25 year old Spinal Muscular Atrophy (SMA) patients. | Spinal Muscular Atrophy (SMA) MedDRA version: 9.1 Level: LLT Classification code 10041582 Term: Spinal muscular atrophy | Product Code: TRO19622 Pharmaceutical Form: Capsule* CAS Number: 22033-87-0 Current Sponsor code: TRO19622 Other descriptive name: 4-cholesten-3-one,oxime Concentration unit: mg milligram(s) Concentration type: range Concentration number: 125-250 | TROPHOS | Authorised | Female: yes Male: yes | Phase 1b | France | ||||
| 69 | EUCTR2005-002822-78-DE | 09/01/2006 | 19 March 2012 | Can treatment with human growth hormone increase strength in spinal muscular atrophy type II and III? - SMA-GH | Can treatment with human growth hormone increase strength in spinal muscular atrophy type II and III? - SMA-GH | Spinal muscular atrophy (SMA) is an autosomal recessive disease. Due to the genetic defect, a molecule called “spinal motor neuron” (SMN) protein is lacking, resulting in muscle weakness. In SMAs muscle weakness is found most often at the level of proximal muscles. The disease is life-threatening and chronically debilitating. | Trade Name: Norditropin SimpleXx Product Code: GH Pharmaceutical Form: Injection* INN or Proposed INN: Somatropin Other descriptive name: Norditropin SimpleXx Concentration unit: mg/ml milligram(s)/millilitre Concentration type: equal Concentration number: 10- Pharmaceutical form of the placebo: Injection* Route of administration of the placebo: Subcutaneous use | Klinik Neuropädiatrie und Muskelkrankheiten | Not Recruiting | Female: yes Male: yes | 20 | Germany | ||||
| 70 | NCT00774423 | January 2006 | 19 February 2015 | Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA) | Multicentric, Randomized, Double-blind Study Versus Placebo, With Two Parallel Groups Treated to Evaluate the Efficacy and the Tolerance of Riluzole in Children and Young Adults (6 to 20 Years of Age) With SMA. (Type II and Type III) | SMA | Drug: Riluzole | Assistance Publique - Hôpitaux de Paris | Not recruiting | 6 Years | 20 Years | Both | 141 | Phase 2/Phase 3 | France | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 71 | NCT00227266 | September 2005 | 19 October 2017 | Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy | Multi-center Phase II Trial of Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (SMA CARNI-VAL Trial) | Spinal Muscular Atrophy | Drug: Valproic Acid and Levocarnitine;Drug: Placebo | University of Utah | Families of Spinal Muscular Atrophy;Leadiant Biosciences, Inc.;Abbott | Not recruiting | 2 Years | 17 Years | All | 94 | Phase 2 | United States;Canada |
| 72 | NCT00568698 | January 2004 | 19 February 2015 | A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients | A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients | Muscular Atrophy, Spinal | Drug: Hydroxyurea | Stanford University | Not recruiting | N/A | 2 Years | Both | 18 | Phase 1/Phase 2 | United States | |
| 73 | NCT00568802 | January 2004 | 16 September 2019 | A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients | A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients | Muscular Atrophy, Spinal | Drug: Hydroxyurea;Drug: Placebo to match hydroxyurea | Stanford University | Not recruiting | 1 Year | 10 Years | All | 27 | Phase 1/Phase 2 | United States | |
| 74 | NCT00374075 | September 2003 | 5 September 2016 | Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy | In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy | Spinal Muscular Atrophy | Drug: Valproic Acid | University of Utah | Families of Spinal Muscular Atrophy;Sigma Tau Pharmaceuticals, Inc.;Abbott | Not recruiting | 2 Years | N/A | Both | 42 | Phase 1 | United States |
| 75 | NCT00004771 | October 1992 | 19 February 2015 | Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease | Spinal Muscular Atrophy;Amyotrophic Lateral Sclerosis;Spinobulbar Muscular Atrophy | Drug: leuprolide;Drug: testosterone | National Center for Research Resources (NCRR) | Ohio State University | Not recruiting | 18 Years | N/A | Male | 40 | Phase 2 |